Ivosidenib in combination with azacitidine receives FDA approval for the treatment of IDH1-mutated newly diagnosed AML

On May 25, 2022, it was announced that the U.S. Food and Drug Administration (FDA) approved the use of ivosidenib tablets in combination with azacitidine for IDH1-mutated acute myeloid leukemia (AML) in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy.

Ivosidenib is a small molecular IDH1 inhibitor, already approved across multiple IDH1-mutated cancer types. This expanded approval follows results from the phase III AGILE trial (NCT03173248), previously summarized in a visual abstract on the AML Hub, in which ivosidenib plus azacitidine demonstrated significantly improved event-free survival, overall survival, and overall response rate compared with azacitidine plus placebo. The safety profile was favorable in both treatment arms, and the most common adverse events were febrile neutropenia, anemia, thrombocytopenia, and pneumonia (22.5%).